FDA issues guidance on pharmacogenomics data

ROCKVILLE, MD., November 3, 2003 -- The FDA has issued a new
document -- Draft Guidance for Industry: Pharmacogenomic Data
Submissions -- that encourages drug and biologic developers to
conduct pharmacogenomic tests during drug development and clarifies
how FDA will evaluate the resulting data.

"Pharmacogenomics holds great promise to shed scientific light
on the often risky and costly process of drug development, and to
provide greater confidence about the risks and benefits of drugs in
specific populations," said FDA Commissioner Mark B. McClellan,
M.D., Ph.D. "Pharmacogenomics is a new field, but we intend to do
all we can to use it to promote the development of medicines. By
providing practical guidance on how to turn the explosion of
pharmacogenomic information into real evidence on new drugs, we are
taking an important step toward that goal."

Pharmacogenomics deals with the small genetic differences that
help explain why some people respond positively to a drug, while
others don't respond, or may experience a side effect. Genetic
differences also can predict variations in drug metabolism-how
quickly or slowly a drug is eliminated from the body. In the draft
guidance, FDA said that the promise of pharmacogenomics lies in its
potential ability to individualize therapy by predicting which
individuals have a greater chance of benefit or risk -- thus
helping to maximize the effectiveness and safety of drugs. FDA
believes that pharmacogenomic testing can be smoothly integrated
into drug development processes.

This is FDA's first step towards integration of this new field
into the process of demonstrating that new drugs are safe and
effective, and thus the regulatory guidance is intended to
facilitate this integration. This guidance is intended to ensure
that evolving regulatory policies and study designs are based on
the best science; provide public confidence in this new field where
scientifically appropriate; facilitate the use of such tests during
drug development; and clarify for industry what types of
pharmacogenomic data to submit to FDA.

"Using genomic testing to guide drug therapy will constitute a
significant shift from the current practice of population-based
treatment towards "fine-tuning" individual therapy," said Janet
Woodcock, FDA's Director of the Center for Drug Evaluation and
Research.

Currently, scientific understanding of phamacogenomics is most
advanced in the drug metabolism area, and early results are
expected in this field. However, FDA anticipates rapid evolution of
additional uses. For example, it is hoped that pharmacogenomic
testing will help identify cancers that have a high probability of
responding to a particular medication or regimen. Pharmacogenomics
may also be used to help track down the cause of certain rare,
serious drug side effects.

The guidance provides specific criteria and recommendations on
submission of pharmacogenomic data to investigational new drug
applications (INDs) and New Drug Applications (NDAs) and Biological
License Applications (BLAs). This includes information on what data
is needed, and how FDA will or will not use such data in regulatory
decisions.

Because there is a need for scientific exchange, the agency is
asking for voluntary submissions of research information. This data
will help FDA gain experience as the field evolves. In these cases,
FDA advises sponsors to clearly label voluntary submissions; and
the agency advises that it will not use information from voluntary
reports for regulatory decisions. If a sponsor subsequently
develops additional data that meet the criteria for submission for
regulatory purposes, the Agency advises sponsors that such data
should be submitted as explained in the guidance.

FDA's Science Board recently (April 2003) endorsed FDA proposals
to move forward with this guidance on assisting industry in
deciding whether a submission is needed. In addition, FDA held
public meetings and workshops in which the key issues for drug
development were identified.